About 35 percent of those attendees will come from outside the United States, said André Singer, project director at Terrapinn, a New York-based events organizer.

Marshall Summar, director of the Rare Disease Institute at Children’s National, will speak at WODC 2019. (Photos by Larry Luxner)

“This is the largest global rare disease conference,” Singer told BioNews Services, which plans to cover WODC 2019 on behalf of this website. “Since its inception in 2011, it has always focused on how to advance rare diseases research and expedite orphan drug development. That’s the commercial focus of the event.”

The CEOs of two of the world’s largest rare disease umbrella groups — Peter Saltonstall of the National Organization for Rare Disorders (NORD), and Yann Le Cam of Paris-based Eurordis — are among the top speakers. Also making presentations will be representatives of the U.S. Food and Drug Administration (FDA), and national rare disease associations for Argentina, Bulgaria, Canada, China, India, Spain, and Russia.

“Access and affordability, which relates to pricing and reimbursement, are the big contentious areas we are tackling this year,” Singer said. “This includes the evolving landscape for advanced therapies and talking about how cell and gene therapies are being developed for rare diseases.”

Yann Le Cam, CEO of Eurordis.

“The contract manufacturers are at overcapacity already to handle the growing demand for cell and gene therapies,” he added. “One of the delivery methods for gene therapy is viral vectors — but this is an evolving field. Batch efficiency and production of those vectors is quite troublesome.”

Two executives at Biogen — André Liamas and Tom Rising — will offer an overview of the global reimbursement landscape for the company’s spinal muscular atrophy (SMA) injectable therapy Spinraza (nusinersen), along with a Brazil case study. Mark Galbraith, head of quality control and analytical sciences at Spark Therapeutics, will talk on “Challenges in analytical development for gene therapy for rare diseases and Luxturna as a case study.”

“Another big focus is patient data, artificial intelligence and digital health,” Singer said, noting a keynote panel on April 11 that includes NORD’s Saltonstall and Le Cam of Eurordis, as well as Simon Kos, chief medical officer of Microsoft; James Greenwood, CEO of the Washington-based Biotechnology Innovation Organization, and Oodaye Shukla of HVH Patient Precision Analytics.

A panel on potential medicines for rare cancers will be headed by Samit Hirawat, head of oncology global development at Novartis. John Maraganore, CEO and director of Alnylam Pharmaceuticals, is to speak on advancing RNAi therapeutics as a whole new class of medicines.

Registration prices before March 1 range from $1,570 for a group booking of three or more attendees for a two-day pass to $2,560 for an individual pass covering all three days. For more information, please click here.

Disclaimer:

Alport Syndrome News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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